Looking for abnormal, clumped features — dubbed mulberry bodies by scientists — in cells of a person’s urine may be…
Articles by Marisa Wexler, MS
ST-920 (isaralgagene civaparvovec), an experimental gene therapy for Fabry disease that’s currently headed toward Phase 3 clinical testing,…
In Fabry disease, a protein called alpha-synuclein forms clumps inside kidney cells, a toxic buildup that isn’t reversed by…
Freeline Therapeutics is pausing development of FLT190, an experimental gene therapy for Fabry disease. The move will…
The same mutation caused both late-onset and classic forms of Fabry disease among different members of the same family,…
Testing family members of people diagnosed with Fabry disease can identify new individuals with the condition and improve their…
The European Commission (EC) is expected to decide whether to approve PRX-102 (pegunigalsidase alfa) for adults with Fabry…
People with Fabry disease often have impairment in the nerves that help regulate heartbeat, which may serve as a…
More than one in 10 people with Fabry disease have a form of swelling called lymphedema, a recent study…
The U.S. Food and Drug Administration (FDA) has placed a hold on the 4D-310 clinical program, an experimental gene therapy…